7月这几款创新药物有望被FDA或欧盟委员会批准 | Bilingual

等应用领域。如您有任何业务需求，叮嘱长按显影下方二维码，或点选文末“阅读原文/Read more”，即可则会面时业务联结的平台，填写业务需求信息。

Expected US and EU New Drug Approvals in July2022In July, EU and US regulatory agencies are each expected to make regulatorydecisions on one potential drug approval, based on their expected PDUFA goal dates and the EMA’sCommittee for Medicinal Products for Human Use (CHMP) recommended approvaldates, respectively. Another 2 innovative drugs have received positive opinions from CHMP and are expected to receive approval decisions in the third quarter (possibly in July if the approval process is fast).Drug: TislelizumabCompany: BeiGene / NovartisIndication(s): Esophagealsquamous cell carcinoma Tislelizumabis a humanized IgG4 monoclonal antibody that targets PD-1, seeking US FDAapproval for the potential treatment of unresectable recurrent locally advancedor metastatic esophageal squamous cell carcinoma (ESCC). In the US, in September 2021 the FDA accepted the BLAfiling by BeiGene on behalf of Novartis for the drug for unresectable recurrentlocally advanced or metastatic ESCC after prior systemic therapy, based onpositive data from the phase 3 RATIONALE 302 trial, with a PDUFA goal dateset as July 12, 2022. ESCC is the most common type of esophageal cancer and thesixth leading cause of cancer-related death globally, with two-thirds of ESCCpatients having advanced or metastatic disease at the time of diagnosis. Ifapproved in the US, it would offer a new option to treat this devastatingdisease, which has limited treatment options hitherto and an average 5-yearsurvival rate of just 5% for the metastatic form.Drug: EladocageneexuparvovecCompany: PTC TherapeuticsIndication(s): AADC deficiencyOne of thetherapeutic candidates anticipating European Commission (EC)’s approval decisionis PTC’s eladocagene exuparvovec (Upstaza), a recombinantadeno-associated virus serotype 2 (AAV2)-based gene therapy containing thehuman DDC gene, designed to correct the underlying genetic defect by deliveringa functioning DDC gene directly into the putamen, as a one-time treatment for aromatic l-amino acid decarboxylase(AADC) deficiency. In May 2022, CHMP gave a positive opinion recommending approval of the gene therapy. AADC deficiency is a fatal rare neurologicaldisorder that causes severe disability with no effective treatment. The companyexpects the EC to grant marketing authorization for the therapy under exceptionalcircumstances in July, approximately 2 months following CHMP’srecommendation. Upon EC approval, eladocagene exuparvovec wouldbe the first disease-modifying treatment for AADC deficiency for patients 18months and older, as well as the first marketed intracerebral genetherapy. It has potential to become the standard of care for the treatment of AADC deficiency.Drug: LenacapavirCompany: Gilead SciencesIndication(s): HIV-1 infectionLenacapavir is a potential "first-in-class" HIV-1capsid inhibitor being developed by Gilead as a sustained-release, 6-monthly,subcutaneous injectable formulation, to use in combination with otherantiretroviral(s), as a potential treatment of multidrug-resistant HIV-1infection in adults for whom it is otherwise not possible to construct asuppressive antiviral regimen. In August 2021, the EMA validated Gilead’s MAAfor lenacapavir shortly after its submission under the centralized licensingprocedure and in June 2022, the agency’s CHMP adopted a positive opinion,recommended granting marketing authorization of the drug in the EU. The MAAfiling is supported by positive data from the phase 2/3 CAPELLA trial whichdemonstrated efficacy, safety and tolerability of the drug, in combination withan optimized background regimen, with an undetectable viral load achieved atweek 26. Gilead expected the final EC decision to be in the third quarter ofthe year. If authorized, lenacapavir could provide a new, long-acting,6-monthly treatment option to this unmet need group with no cure and limitedtreatment choices so far. Gilead is also seeking approval of the drug in the USfor the same indication.Drug: Valoctocogene roxaparvovecCompany: BioMarin PharmaceuticalIndication(s): Hemophilia AAnother drug candidate pending regulatoryapproval in the EU is BioMarin’s valoctocogene roxaparvovec (Roctavian), aninvestigational adeno-associated virus serotype 5 (AAV5) gene therapy for thepotential treatment of severe hemophilia A. The MAA filing was resubmitted in June 2021 with the supplementation ofone-year safety and efficacy data from the phase 3 GENEr8-1 trial. Thetherapy was granted accelerated assessment and the EMA’s CHMP adopted apositive opinion in June 2022, recommending conditional marketing approval(CMA) of the gene therapy based on totality of data from the extensive clinicaldevelopment program, including 2-year outcomes from the global GENEr8-1 study,as well as up to 5 years of follow-up in the ongoing phase 1/2 dose escalationstudy, demonstrating substantial and sustained reduction in bleeding. BioMarinanticipates EC’s final decision in 3Q 2022 with launch of the product by theend of the year. It isestimated that more than 20,000 adults across Europe, Middle East and Africaare affected by severe hemophilia A and the standard of care for this patientgroup is chronic lifelong injectable therapy. Upon approval, Roctavian wouldbecome the first gene therapy in Europe for hemophilia A and could potentiallybe the standard of care therapy for severe hemophilia A. 了解更多FDA获批本品

叮嘱点选下面则会面时我们的小程序

参考文献：[1] Novartisexpands Oncology pipeline with in-licensing of tislelizumab from BeiGene.Retrieved June 28, 2022 from, [2] JP Morgan40th Annual Healthcare Conference - January 11, 2022. Retrieved June 28, 2022 from, ir-api.eqs.com/redirect/5b3a2f61-fffb-40e7-b514-19e3da2ecf73[3] China NMPAApproves Tislelizumab for Patients with Second-Line Esophageal Squamous CellCarcinoma. Retrieved June 28, 2022 from, / [4] China NMPAApproves Tislelizumab for Recurrent or Metastatic Nasopharyngeal Cancer.Retrieved June 28, 2022 /[5] Novartisannounces first FDA filing acceptance for anti-PD-1 antibody tislelizumab forpeople with esophageal cancer. Retrieved June 28, 2022 from, [6] ArcutisPipeline - Topical Roflumilast Cream. Retrieved June 28, 2022 from, [7] AstraZenecato strengthen therapy area franchise through acquisition of Takeda’srespiratory business. Retrieved June 28, 2022 from, # [8] FDA AcceptsArcutis Biotherapeutics’ New Drug Application for Roflumilast Cream for Adultsand Adolescents with Plaque Psoriasis. Retrieved June 28, 2022 from, / [9] PTCTherapeutics Provides Corporate Update and Highlights Pipeline Progress at 2020J.P. Morgan Healthcare Conference. Retrieved June 28, 2022 from, ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-provides-corporate-update-and-highlights[10] PTC TherapeuticsReceives Positive CHMP Opinion for Upstaza™ for the Treatment of AADCDeficiency. Retrieved June 28, 2022 from, ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-receives-positive-chmp-opinion-upstazatm[11] European MedicinesAgency Validates Gilead’s Marketing Authorization Application for Lenacapavir,an Investigational, Long-Acting Capsid Inhibitor for the Treatment of HIV-1 inPeople With Limited Therapy Options. Retrieved June 28, 2022 from, [12] InvestigationalLenacapavir Receives Positive CHMP Opinion for People With Multi-Drug ResistantHIV. Retrieved June 28, 2022 from, [13] Gilead Resubmits New Drug Application ToU.S. Food And Drug Administration For Lenacapavir, An Investigational,Long-Acting HIV-1 Capsid Inhibitor. Retrieved June 28, 2022 from, Food-and-Drug-Administration-for-Lenacapavir-an-Investigational-Long-Acting-HIV-1-Capsid-Inhibitor/default.aspx[14] BioMarinReceives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec GeneTherapy to Treat Adults with Severe Hemophilia A. Retrieved June 28, 2022 from,investors.biomarin.com/2022-06-24-BioMarin-Receives-Positive-CHMP-Opinion-in-Europe-for-Valoctocogene-Roxaparvovec-Gene-Therapy-to-Treat-Adults-with-Severe-Hemophilia-A[15] BioMarinSubmits Marketing Authorization Application to European Medicines Agency forValoctocogene Roxaparvovec to Treat Severe Hemophilia A. Retrieved June 28,2022 from, [16] BioMarinResubmits Marketing Authorization Application (MAA) to European MedicinesAgency for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A. RetrievedJune 28, 2022 from, investors.biomarin.com/2021-06-28-BioMarin-Resubmits-Marketing-Authorization-Application-MAA-to-European-Medicines-Agency-for-Valoctocogene-Roxaparvovec-to-Treat-Severe-Hemophilia-A[17] BioMarinPharmaceutical Inc. (BMRN) Q1 2022 Earnings Call Transcript. Retrieved June 28,2022 from, / 免责声明：药明黑格尔内容团队专注概述世界性生物技术肥胖研究进展。本文仅作信息交流之目的，原文论据不代表药明黑格尔看法，亦不代表药明黑格尔反对或反对原文论据。本文也不是疗程拟议推荐。如需获疗程拟议范本，叮嘱赶赴正规医院就诊。

著作权解释：本文来自药明黑格尔内容团队，热烈欢迎个人发信至朋友圈，谢绝新闻媒体或机构未经授权以任何基本概念发表文章至其他的平台。发表文章授权叮嘱在「药明黑格尔」搜狐公众号回复“发表文章”，获取发表文章一览表。

分享，点赞，在看，聚焦世界性生物技术肥胖创新

。

重庆看男科的专业医院
株洲看妇科去哪个医院好
南京看妇科哪个医院好
郑州哪个妇科医院好
杭州哪的妇科医院好